Combining genomics, drug sensitivity testing could be ‘invaluable’ cancer treatment tool
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Key takeaways:
- A functional precision medicine approach produced significant objective response, PFS in children with cancer.
- Results from drug sensitivity testing came back within 2 weeks.
An innovative combination of drug sensitivity testing and genomics helped identify therapeutic options for children with relapsed or refractory solid or hematologic malignancies, according to results published in Nature Medicine.
Patients who received therapies guided with the functional precision medicine (FPM) platform had a significantly superior objective response rate than those who had nonguided treatments. Additionally, those therapies produced PFS 1.3-times greater (median, 8.5-fold) than their previous treatments.
“Cancer, especially advanced cancer, is an individualized disease and should be treated as such,” Diana J. Azzam, PhD, assistant professor at Florida International University, told Healio. “FPM is a path to truly personalized cancer treatments, helping patients get the right drug at the right time.”
Researchers enrolled 25 children (median age, 10 years;84% white; 68% Hispanic) between 2019 and 2022 with various cancer types who had at least two previous treatments.
They completed drug sensitivity testing on 88% of patients who provided tissue samples and had results back in a median of 9 days for hematologic cancers and 10 for solid tumors.
Clinicians performed FPM-guided treatments on six patients, 83% of whom had an objective response. Of eight patients who received nonguided therapies, just 13% had a response.
Healio spoke with Azzam about the trial, FPM and its future impact.
Healio: Why did you and your colleagues feel it was important to conduct this study?
Azzam: Even in 2024, one in three patients with cancer still die of their disease. These patients are failed by the standard-of-care treatment protocols and end up trapped in cycles of trial-and-error treatments to find effective options. This represents a significant unmet clinical need, with patients and doctors desperate for new technologies to help patients find the right drug at the right time. My colleagues and I believe that personalized drug sensitivity testing can help patients and doctors find effective tailored treatment options.
Healio: Can you describe FPM?
Azzam: It combines today’s clinical genomics tools with cutting-edge drug sensitivity testing on the patient’s own cancer cells. Drug sensitivity testing is a process by which a library of anticancer drugs is tested against tumor tissue samples from a patient to determine which drugs effectively kill that patient’s cancer cells. While genomic tumor profiling has provided clinical benefit to numerous patients, many do not have actionable mutations or cannot access treatments matched to the mutations they have. Drug sensitivity testing matches patients to treatment options beyond those linked to specific mutations, including drugs with limited information on predictive mutations. This provides patients and physicians with personalized data to validate and rank multiple treatment options tailored to that patient, beyond what genomics testing can provide.
Healio: What do you consider to be the most important clinical implication of your trial?
Azzam: Personalized drug sensitivity testing can be provided as fast as, if not faster than, genomic tumor testing, and that combining drug sensitivity testing with genomic tumor profiling identifies treatment options that can provide significant clinical benefit to patients with even advanced cancer.
Healio: How can physicians use this study to better serve their patients?
Azzam: This study shows that new approaches to oncology are possible and can provide significant benefit to patients, even if they’ve exhausted standard treatment options. Right now, we are getting [Clinical Laboratory Improvement Amendments (CLIA) certification for our drug sensitivity testing approach, which enables us to provide treatment options to patients outside of a clinical trial. Accessing FPM data to support treatment decision-making, especially when doctors might otherwise be forced to take a trial-and-error approach, is one of most direct ways this study can help doctors serve their patients. We believe this will be an invaluable clinical tool for patients and doctors going forward.
Healio: Are there any important questions that must be answered in future research?
Azzam: The biggest open question right now is how great an impact our FPM approach can have. We have shown it is possible to return data quickly, and that patients can benefit significantly from FPM-guided treatments. Our first study was in 25 patients. Now we need to see what happens when 250 patients have FPM-guided options. We also need to explore how FPM can benefit patients if we provide it earlier in the clinical treatment cycle rather than as a last resort.
For more information:
Diana J. Azzam, PhD, can be reached at dazzam@fiu.edu.
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